A few hours after Harry (not his real name) was born, he became restless and did not want to be breastfed. His mother notice that his left arm and leg were shaking rhythmically and something was not right.
Harry was immediately transfer to the neonatal intensive care unit.
An MRI scan reveal that he had suffer a severe stroke.
Doctors told Harry’s parents that there was no treatment they could give the child.
Harry would probably be disable.
Most people think of stroke as something that mainly affects the elderly, but it can also occur in newborn babies.
These “perinatal strokes” happen when one of the major arteries to the brain becomes block, leading to a lack of blood supply and hence oxygen to certain brain areas.
About one in 5,000 newborns have a stroke.
It usually happens in the first few days after they are born.
Most of the babies will have problems later in life, with the severity of the problems depending on which brain areas were injured.
These problems can include muscle tightness in the arms and legs (cerebral palsy), behaviour problems, learning difficulties and epilepsy.
No therapy exists for newborns with stroke.
Researchers, including our own team at University Medical Center Utrecht, have working on new treatments, one of which involves stem cells.
Stem cells have the ability to turn into many different cells in the body, and they are little factories of several growth factors (proteins that stimulate the growth of specific tissues).
The theory is that if we can get stem cells into the damage part of a baby’s brain, the stem cells’ growth factors will stimulate the brain to repair itself.
Effective in animals Earlier studies in animals show that injecting stem cells into the brains of newborn mice with stroke dramatically reduce the amount of brain damage and disability they suffer.
The experiments show that the treatment was safe and had no side-effects in the mice.
These animal studies gave us hope that the treatment would work in newborn babies, too, preventing a lifetime of disability.
But how do you deliver stem cells to a baby’s brain without having to use needles or surgery?
It decide to try an intranasal route (through the nose), which was tested in mice.
After it deliver the stem cells intranasally, the cells travel rapidly and specifically to the injured brain areas.
The injure brain area sends out “alarm signals” which guide the stem cells to the right spot in the brain.
When the stem cells arrive at the damaged area, they secreth growth factors that boost the repair systems of the mice’s brains.
Within a few days, the stem cells were broken down and not traceable in the brain any longer.
After many experiments with this method, we conclude that dripping stem cells in the nose is the safest and most efficient way to deliver them to the brain.
Ten babies After many years of laboratory research, they have finally test the treatment in babies.
The results have published in The Lancet Neurology.
Baby Harry was the first baby to participate in the study and receive stem cells within a week of being born.
To ask parents to enroll in an experimental therapy in the first week of their newborn child’s life is a very delicate process.
After had a long conversation with his parents, they decide to let their son take part in the study.
Harry receive stem cells via nose droplets, a procedure that took only several minutes.
Then, Harry was monitor closely for a few days before he went home.
They treated ten newborns who were transfer from hospitals across the Netherlands to the University Medical Center Utrecht after suffering from a stroke.
In all ten newborns, the stem cell droplets were administer without any complications.
There was one baby who had a mild fever after the treatment, which quickly clear up on its own.
A follow-up MRI scan of the brain made three months after the stroke show less injury than expect, possibly because of the stem cells.
At four months, the treat babies, including Tom, perform well when the quality of their movements was test.
When the children are two years old, we will check their development again.
They are now looking for opportunities to proceed with a randomise control trial (the gold standard for medical studies) to prove that stem cell therapy can effectively repair brain injury after perinatal stroke.
The discovery of a new and safe therapy with stem cells also opens up opportunities for other babies with brain injury, such as babies who are born too early, or babies that suffer from a lack of oxygen during birth (perinatal asphyxia).
Stem cell therapy gives hope to the most vulnerable patient group, with possible lifelong benefits.